@FidelisAurelius Agree management seems a bit lackluster. Validation in FSHD would be such a huge signal for additive muscle regen capacity independent of dystrophin deficiency... How much would professional athletes (or their teams) pay for a faster recovery timeline in msk related injuries...
@Banana_Oncology I knew someone who worked in global health there and from what I recall rural communities, which constitutes a sizeable % of the population (who suffered the most from HBV/HCC), still have really poor access to this day
@anthonystaj While it's almost certainly a lock for rejection, could you imagine the regulatory precedent if this obfuscative garbage from CAPR stood...
Yea sure, feel free to p-hack your SAP 3 times after all data has been collected include one procrastinated night before amendment.
@augurbio DTIL is very interesting. Slightly unsure of unmet need in HBV given entecavir is cheap and is basically a functional cure, however in the DMD space they still remains of the most underrated names
this is genuinely horrible. lobbyists are winning in DC.
@steveusdin1 not really sure how telling the public why a CRL was issued is a bad thing! transparently disclosing the precise logic can help all stakeholders hold sponsors accountable. investors can see whether companies are being honest, patients can see whether the BLA/NDAs are truly ready for primetime (and to be used in their body), and other drug developers can see if mistakes ought to be avoided and speed up the next live saving therapy!
genuinely confused how this is a problem given that many companies are less than forthcoming with their public disclosures
@plainyogurt21@clinmean Are you questioning whether or not they can hit stat-sig at all? Or meet/approach dupixent benchmark ~ 60%.
Oral suspension corticosteroid (budesonide) hit 53%
@plainyogurt21 Likely the latter to avoid sampling risk issues with very low n arms? Absolute PEC % change is in line with (slightly below) dupi. EoE-HSS is also in line.
Some questions around validity of <6 benchmark, ie Benralizumab 88% meeting <6 without mitigation of clinical symptoms
@maxmarchione The company that failed a n=140k trial assessing whether or not their screening identified any meaningful additional cases of earlier stage cancers? 🤔